AU2012353330B2 - Chimeric double-stranded nucleic acid - Google Patents
Chimeric double-stranded nucleic acid Download PDFInfo
- Publication number
- AU2012353330B2 AU2012353330B2 AU2012353330A AU2012353330A AU2012353330B2 AU 2012353330 B2 AU2012353330 B2 AU 2012353330B2 AU 2012353330 A AU2012353330 A AU 2012353330A AU 2012353330 A AU2012353330 A AU 2012353330A AU 2012353330 B2 AU2012353330 B2 AU 2012353330B2
- Authority
- AU
- Australia
- Prior art keywords
- modified
- nucleic acid
- base
- rna
- pct
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
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Classifications
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/15—Nucleic acids forming more than 2 strands, e.g. TFOs
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
- C12N2310/3181—Peptide nucleic acid, PNA
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/341—Gapmers, i.e. of the type ===---===
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
- C12N2310/3513—Protein; Peptide
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
- C12N2310/3515—Lipophilic moiety, e.g. cholesterol
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/50—Physical structure
- C12N2310/53—Physical structure partially self-complementary or closed
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2320/00—Applications; Uses
- C12N2320/50—Methods for regulating/modulating their activity
- C12N2320/52—Methods for regulating/modulating their activity modulating the physical stability, e.g. GC-content
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
JP2011275488 | 2011-12-16 | ||
JP2011-275488 | 2011-12-16 | ||
PCT/JP2012/083180 WO2013089283A1 (en) | 2011-12-16 | 2012-12-17 | Chimeric double-stranded nucleic acid |
Publications (2)
Publication Number | Publication Date |
---|---|
AU2012353330A1 AU2012353330A1 (en) | 2014-07-31 |
AU2012353330B2 true AU2012353330B2 (en) | 2018-04-19 |
Family
ID=47559626
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
AU2012353330A Active AU2012353330B2 (en) | 2011-12-16 | 2012-12-17 | Chimeric double-stranded nucleic acid |
Country Status (9)
Country | Link |
---|---|
US (5) | US9816089B2 (zh) |
EP (1) | EP2791335B1 (zh) |
JP (7) | JP6112569B2 (zh) |
CN (2) | CN112386605A (zh) |
AU (1) | AU2012353330B2 (zh) |
CA (2) | CA3106312A1 (zh) |
ES (1) | ES2707232T3 (zh) |
IN (1) | IN2014CN04590A (zh) |
WO (1) | WO2013089283A1 (zh) |
Families Citing this family (46)
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BR112012000828A8 (pt) | 2009-07-06 | 2017-10-10 | Ontorii Inc | Novas pró-drogas de ácido nucleico e métodos de uso das mesmas |
US10428019B2 (en) | 2010-09-24 | 2019-10-01 | Wave Life Sciences Ltd. | Chiral auxiliaries |
RU2014105311A (ru) | 2011-07-19 | 2015-08-27 | Уэйв Лайф Сайенсес Пте. Лтд. | Способы синтеза функционализованных нуклеиновых кислот |
CA3106312A1 (en) * | 2011-12-16 | 2013-06-20 | National University Corporation Tokyo Medical And Dental University | Chimeric double-stranded nucleic acid |
JP6268157B2 (ja) | 2012-07-13 | 2018-01-24 | 株式会社Wave Life Sciences Japan | 不斉補助基 |
WO2014132671A1 (en) * | 2013-03-01 | 2014-09-04 | National University Corporation Tokyo Medical And Dental University | Chimeric single-stranded antisense polynucleotides and double-stranded antisense agent |
WO2014148620A1 (ja) * | 2013-03-21 | 2014-09-25 | 国立大学法人東京医科歯科大学 | 二重鎖核酸結合剤、当該結合剤-二重鎖核酸複合体、当該複合体を含有する医薬品組成物、及び、当該複合体の製造方法 |
US10190117B2 (en) * | 2013-06-16 | 2019-01-29 | National University Corporation Tokyo Medical And Dental University | Double-stranded antisense nucleic acid with exon-skipping effect |
WO2015105083A1 (ja) * | 2014-01-07 | 2015-07-16 | 塩野義製薬株式会社 | アンチセンスオリゴヌクレオチド及び糖誘導体を含む二本鎖オリゴヌクレオチド |
US9714288B2 (en) | 2014-09-30 | 2017-07-25 | The Regents Of The University Of California | Antisense compounds and uses thereof |
US11116843B2 (en) | 2015-09-25 | 2021-09-14 | Ionis Pharmaceuticals, Inc. | Conjugated antisense compounds and their use |
EP3366773A4 (en) * | 2015-10-23 | 2019-06-12 | Rena Therapeutics Inc. | NUCLEIC ACID COMPLEX |
EP3366774A4 (en) * | 2015-10-23 | 2019-03-27 | Rena Therapeutics Inc. | NUCLEIC ACID COMPLEX WITH AT LEAST ONE WOOD STRUCTURE |
JP6950958B2 (ja) * | 2015-12-01 | 2021-10-13 | 国立大学法人 東京医科歯科大学 | 核酸を含む経腸投与用組成物 |
CN116064544A (zh) * | 2016-01-26 | 2023-05-05 | 日产化学株式会社 | 单链寡核苷酸 |
MA45328A (fr) * | 2016-04-01 | 2019-02-06 | Avidity Biosciences Llc | Compositions acide nucléique-polypeptide et utilisations de celles-ci |
WO2018056442A1 (ja) * | 2016-09-23 | 2018-03-29 | 国立大学法人東京医科歯科大学 | 血液脳関門通過型ヘテロ2本鎖核酸 |
EP3521430A4 (en) | 2016-09-29 | 2020-05-20 | National University Corporation Tokyo Medical and Dental University | DOUBLE-STRANDED NUCLEIC ACID COMPLEX WITH OVERHEAD |
CA3052801A1 (en) * | 2017-02-06 | 2018-08-09 | Nissan Chemical Corporation | Single-stranded oligonucleotide |
US11638717B2 (en) | 2017-03-29 | 2023-05-02 | Shionogi & Co., Ltd. | Complex of nucleic acid medicine and multibranched lipid |
WO2018180005A1 (ja) * | 2017-03-31 | 2018-10-04 | 国立研究開発法人日本医療研究開発機構 | コンドロイチン硫酸生合成を阻害するアンチセンス核酸 |
EP3647423A4 (en) * | 2017-06-30 | 2021-03-24 | National University Corporation Tokyo Medical and Dental University | DOUBLE STRANDED HETERO-ANTI-MIRNA |
US11555188B2 (en) | 2017-07-26 | 2023-01-17 | Nissan Chemical Corporation | Single-stranded oligonucleotide |
US11674141B2 (en) | 2018-02-28 | 2023-06-13 | National University Corporation Tokyo Medical And Dental University | Ischemic-lesion-site-specific gene therapy |
JP7333079B2 (ja) | 2018-03-19 | 2023-08-24 | 国立大学法人 東京医科歯科大学 | 毒性が軽減した核酸 |
WO2019182294A1 (ko) * | 2018-03-19 | 2019-09-26 | 주식회사 케이티 | 비디오 신호 처리 방법 및 장치 |
AU2019237599A1 (en) | 2018-03-20 | 2020-11-12 | Nissan Chemical Corporation | Antisense oligonucleotide having reduced toxicity |
JP7262816B2 (ja) * | 2018-03-22 | 2023-04-24 | 国立大学法人 東京医科歯科大学 | ヘテロ核酸のbbb通過脂質リガンド |
CN113614232A (zh) * | 2019-01-18 | 2021-11-05 | 马萨诸塞大学 | 动态药代动力学修饰锚 |
JPWO2020209285A1 (zh) * | 2019-04-08 | 2020-10-15 | ||
US20220267778A1 (en) | 2019-07-30 | 2022-08-25 | Shionogi & Co., Ltd. | Nucleic acid drug targeting murf1 |
JPWO2021070959A1 (zh) * | 2019-10-11 | 2021-04-15 | ||
EP4090744A4 (en) * | 2020-01-17 | 2024-04-10 | Anastasia Khvorova | UNIVERSAL DYNAMIC PHARMACOKINETIC MODIFYING ANCHORS |
EP4098747A1 (en) | 2020-01-31 | 2022-12-07 | Sanwa Kagaku Kenkyusho Co., Ltd. | Antisense oligonucleotide of atn1 |
EP4116419A1 (en) | 2020-03-04 | 2023-01-11 | Nissan Chemical Corporation | Antisense oligonucleotide of calm2 |
WO2021187392A1 (ja) * | 2020-03-16 | 2021-09-23 | 国立大学法人東京医科歯科大学 | モルホリノ核酸を含むヘテロ核酸 |
CN115968374A (zh) | 2020-06-15 | 2023-04-14 | Liid制药股份有限公司 | 交联型核苷及核苷酸 |
CA3202708A1 (en) | 2020-11-23 | 2022-05-27 | Alpha Anomeric Sas | Nucleic acid duplexes |
US20240141414A1 (en) * | 2021-02-26 | 2024-05-02 | Northwestern University | Cell-free biosensors with dna strand displacement circuits |
WO2022250050A1 (ja) * | 2021-05-25 | 2022-12-01 | 国立大学法人東京医科歯科大学 | scpBNA又はAmNAを含むヘテロ核酸 |
EP4353266A1 (en) | 2021-05-31 | 2024-04-17 | Rena Therapeutics Inc. | Ligand-bound nucleic acid complex |
JPWO2023022229A1 (zh) | 2021-08-19 | 2023-02-23 | ||
JPWO2023026994A1 (zh) | 2021-08-21 | 2023-03-02 | ||
WO2023042876A1 (ja) * | 2021-09-15 | 2023-03-23 | 国立大学法人東京医科歯科大学 | 2'-修飾ヌクレオシドを含むヘテロ核酸 |
WO2023105898A1 (ja) * | 2021-12-07 | 2023-06-15 | リードファーマ株式会社 | アンチセンスオリゴヌクレオチド複合体 |
GB202215614D0 (en) | 2022-10-21 | 2022-12-07 | Proqr Therapeutics Ii Bv | Heteroduplex rna editing oligonucleotide complexes |
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WO2005113571A2 (en) * | 2004-05-13 | 2005-12-01 | The University Of North Carolina At Chapel Hill | Methods for the delivery of oligomeric compounds |
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2012
- 2012-12-17 CA CA3106312A patent/CA3106312A1/en active Pending
- 2012-12-17 AU AU2012353330A patent/AU2012353330B2/en active Active
- 2012-12-17 CN CN202011292547.5A patent/CN112386605A/zh active Pending
- 2012-12-17 WO PCT/JP2012/083180 patent/WO2013089283A1/en active Application Filing
- 2012-12-17 CN CN201280062110.XA patent/CN104126010B/zh active Active
- 2012-12-17 ES ES12815856T patent/ES2707232T3/es active Active
- 2012-12-17 EP EP12815856.5A patent/EP2791335B1/en active Active
- 2012-12-17 CA CA2859581A patent/CA2859581C/en active Active
- 2012-12-17 IN IN4590CHN2014 patent/IN2014CN04590A/en unknown
- 2012-12-17 JP JP2014528740A patent/JP6112569B2/ja active Active
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2014
- 2014-06-13 US US14/303,989 patent/US9816089B2/en active Active
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2017
- 2017-03-08 JP JP2017044034A patent/JP6416301B2/ja active Active
- 2017-10-05 US US15/725,845 patent/US10337006B2/en active Active
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2018
- 2018-06-28 US US16/021,165 patent/US10329567B2/en active Active
- 2018-10-03 JP JP2018188022A patent/JP6638923B2/ja active Active
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2019
- 2019-05-13 US US16/410,803 patent/US11034955B2/en active Active
- 2019-12-12 JP JP2019224279A patent/JP6960123B2/ja active Active
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2020
- 2020-10-27 JP JP2020179738A patent/JP7174384B2/ja active Active
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2021
- 2021-05-27 US US17/332,249 patent/US20210340540A1/en active Pending
- 2021-08-24 JP JP2021136038A patent/JP2022000426A/ja active Pending
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2022
- 2022-09-15 JP JP2022146970A patent/JP7423015B2/ja active Active
Patent Citations (1)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
WO2005113571A2 (en) * | 2004-05-13 | 2005-12-01 | The University Of North Carolina At Chapel Hill | Methods for the delivery of oligomeric compounds |
Non-Patent Citations (1)
Title |
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JEKERLE V ET AL, "Functional comparison of single- and double-stranded mdr1 antisense oligodeoxynucleotides in human ovarian cancer cell lines", JOURNAL OF PHARMACY AND PHARMACEUTICAL SCIENCES, (2005), vol. 8, no. 3, pages 516 - 527 * |
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